Regulatory Aspects of Gene Therapy and Cell Therapy Products A Global Perspective

This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization.

This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs. 

Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure and therapeutic use of these innovative products.  More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, as well as pertinent ‘must-know’ guidelines and regulations. 

Regulatory Aspects of Gene Therapy and Cell Therapy Products: a Global Perspective is part of the American Society of Gene & Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Introduction (MCG and FN).- Updates on ATMP regulations from EU: experience with PRIME procedure and list of approved ATMP.- EU portal for clinical trials application.- European Pharmacopoeia texts on gene therapy medicinal products.- Updates on ATMP regulations from UK after Brexit and list of approved ATMP.- Updates on ATMP regulations from Switzerland and list of approved ATMP.- Updates on ATMP regulations from US FDA: experience with breakthrough/accelerated procedures and list of approved ATMP.- Updates on ATMP regulations from Canada and list of approved ATMP.- Updates on ATMP regulations from Brasil and list of approved ATMP.- Updates on ATMP regulations from Singapore and list of approved ATMP.- Updates on ATMP regulations from Japan: experience with new law and list of approved ATMP.- Updates on ATMP regulations from South Korea and list of approved ATMP.- Updates on ATMP regulations from Taiwan and list of approved ATMP.- Updates on ATMP regulations from Thailand and listof approved ATMP.- ATMP regulations in China and list of approved ATMP.- ATMP regulations in Australia and list of approved ATMP.- ATMP regulations in India and list of approved ATMP.- International harmonization programs.

Maria Cristina Galli was co-editor of the first edition.

Dr. Galli holds a University degree in Biological Sciences and a PhD in Molecular Medicine. Her main expertise is in regulatory sciences for translational medicine in the Advanced Therapy Medicinal Products (ATMP) field, supported by scientific education and more than 20 years research experience in experimental oncology, cellular biology, and molecular immunology.

Until her retirement end of 2020, dr. Galli was senior researcher at the Istituto Superiore di Sanità, Roma, Italy. For 25 years she served as quality assessor for gene therapy and biotechnology medicines in national as well as European procedures; at the same time, she was a Good Manufacturing Practice (GMP) and Good Laboratory Practice (GLP) inspector in the field of gene therapy and biotechnology with special experience in ATMP production and preclinical studies. Dr. Galli was a member of the Committee for Advanced Therapies (CAT) at the European Medicine Agency (EMA) in 2009–2011 and vice-chair/chair of CAT-EMA Gene Therapy Working Party in 2008–2012, in which she participated since its first meeting in early 2000s. Until 2020 she was chair of the ATMP platform in the European infrastructure for translational medicine EATRIS-ERIC, after having been co-chair for 3 years. From 2018 to 2020 she was chair of the Gene Therapy Products Working Party of the European Pharmacopoeia Commission, European Department for the Quality of Medicines, Strasbourg, France, after having served as expert in the previous term (2004-2010) of that working party.

Since 2005 dr. Galli has been regularly invited at international scientific conferences or meetings and has also regularly given classes in master courses at various Italian Universities, on scientific and regulatory aspects of ATMP development and on GMP for ATMP.

Dr. Galli is presently a free-lance consultant in the field of gene and cell therapy development and production.